Duchenne Muscular DystrophyPhase 2NCT02740972

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase 2 study testing NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 7 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Read more about this study

Who is it for?: Ages 4 to 9 years, men only
Conditions the study is for: Duchenne Muscular Dystrophy
What is being tested?: NS-065/NCNP-01
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 16 participants
Where: 7 sitesin 2 countries
Study dates: Dec 2016to Apr 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 4 to 9 years
Men only
No healthy volunteers
Child

Who may be able to join

Male ≥ 4 years and <10 years of age
Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame;
Able to walk independently without assistive devices;
Ability to complete the time to stand, time to run/walk and time to climb assessments;
Stable dose of glucocorticoid for at least 3 months

Who cannot join

Acute illness within 4 weeks prior to the first dose of study medication;
Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
Severe allergy or hypersensitivity to medications;
Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
Patient is taking any other investigational drug currently or within 3 months prior to the start of study treatment; or
Patient has had surgery within the 3 months prior to the first anticipated administration of study medication or surgery is planned for anytime during the duration of the study;
Patient has previously participated in this study or any other study during which NS-065/NCNP-01 was administered.

Trial locations and contacts

7 sites in 2 countries.

Country

City, postcode or hospital

7 sites in 2 countries

United States6 sites

Lurie Children's Hospital
Chicago, Illinois
Duke University Medical Center
Durham, North Carolina
University of Florida Health
Gainesville, Florida
Children's Hospital of Richmond at VCU
Richmond, Virginia
UC Davis
Sacramento, California, 95817
Washington University
St Louis, Missouri

Other trials in United States

Canada1 site

Alberta Children's Hospital
Calgary, Alberta

Other trials in Canada

About this study

Official title: A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Read the detailed description

This is a Phase II, multiple center, 2-period, randomized, placebo-controlled, dose finding study of NS-065/NCNP-01 administered by infusion once weekly for 24 weeks to ambulant boys ages 4-<10 years with DMD. Two dose level cohorts will be enrolled. Period 1 of this study will be conducted in a double-blind fashion. Randomized patients will receive weekly IV infusions of NS-065/NCNP-01 or placebo for the first 4 weeks of their participation (Period 1) and NS-065/NCNP-01 by IV infusion for weeks 5-24 (20 weeks of active treatment - Period 2). Analysis of safety data from Period 1 of the 40mg/kg dose cohort will be completed prior to enrolling patients in the 80mg/kg dose cohort.

Patients completing the 24-week study will be eligible for an open-label extension study.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT), time to stand (TTSTAND), time to run/walk 10 meters (TTRW), time to climb 4 stairs (TTCLIMB) and quantitative muscle testing (QMT). All patients will undergo a muscle biopsy of the bicep at baseline and a second muscle biopsy at Week 24.

Safety will be assessed through the collection of adverse events (AEs), blood and urine laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.

Serial blood samples will be taken at four of the study visits to assess the pharmacokinetics of the study drug.

Conditions: Duchenne Muscular Dystrophy
Being tested: NS-065/NCNP-01, Placebo
Medical subject headings: Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NS-065/NCNP-01Drug
PlaceboDrug

Study groups in NCT02740972
Study group	Role	What they receive
NS-065/NCNP-01 40mg/kg	Experimental	Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 40mg/kg dose once a week for 24 weeks Receives: Drug: NS-065/NCNP-01
NS-065/NCNP-01 80mg/kg	Experimental	Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 80mg/kg once a week for 24 weeks Receives: Drug: NS-065/NCNP-01
Placebo	Placebo comparator	Two patients in each of the dose groups will be administered placebo as an intravenous infusion once a week for 4 weeks followed by 20 weeks of open label treatment Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of Adverse Events as Assessed by CTCAE v4.0.
Treatment emergent adverse events (TEAEs) were summarized for Period 1 by comparing low dose to high dose to placebo and for Period 2 between the low dose cohort and the high dose cohort. TEAEs were summarized both at the patient level for number of TEAEs, highest severity, relationship, action and outcome and at the TEAE level (summarizing events) by organ system and preferred term TEAE as well as severity, relationship, action and outcome.
The Medical Dictionary for Regulatory Activities (MedDRA) version 20.1 was used and the Common Terminology Criteria for Adverse Events (CTCAE) grading.
Measured: 24 weeks of treatment
Dystrophin Production by Western Blot
Percentage normal dystrophin production in muscle biopsies from study participants at baseline and after 24 weeks treatment was measured by Western blot.
To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. Dystrophin protein levels were assessed using standard curves on each gel (range from 0-25% of normal levels) generated by mixing 5 normal control samples with one DMD sample.
Measured: Baseline and 24 weeks of treatment

Secondary outcomes (12)

Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity
The alteration of mRNA splicing was measured by RT-PCR of dystrophin mRNA transcripts from a patient muscle biopsy at baseline and after 24 weeks treatment.
To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. For RT-PCR, bands corresponding to specific versions of the spliced dystrophin mRNA were visualized by gel electrophoresis, and the amounts of different mRNA isoforms were compared.
Measured: Baseline and 24 weeks of treatment
Dystrophin Production by Mass Spectrometry
The production of dystrophin protein was measured by stable isotope mass spectrometry methods from a patient muscle biopsy at baseline and after 24 weeks treatment.
To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity.
Dystrophin peptides were identified and quantified using reversed-phase nanoflow high-performance liquid chromatography with high resolution Mass Spectrometry.
Measured: Baseline and 24 weeks of treatment
Dystrophin Production by Immunofluorescence
The production of dystrophin protein was measured by immunofluorescence staining methods from a patient muscle biopsy at baseline and after 24 weeks treatment.
To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity.
Immunofluorescence staining for dystrophin was performed on serial muscle biopsy sections in duplicate.
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
A secondary efficacy endpoint was compared to Pre-Infusion Visit: quantitative muscle testing (QMT).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT).
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Six-Minute Walk Test (6MWT).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB).
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Climb 4 Stairs (TTCLIMB).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Run/Walk 10 Meters (TTRW).
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity.
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Stand (TTSTAND)
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND)
Measured: Baseline and 24 weeks of treatment
Change From Baseline in Time to Stand (TTSTAND) Velocity
A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND).The results were converted into velocity (rise/time).
Measured: Baseline and 24 weeks of treatment
Change From Baseline in North Star Ambulatory Assessment (NSAA) Score.
The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Measured: Baseline and 24 weeks of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2016Apr 2018

First posted15 Apr 2016 (estimated)
Study startDec 2016
Primary completionMar 2018
Study completionApr 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02740972
Sponsor's protocol ID: NS-065/NCNP-01-201
Status last verified: Nov 2021
First submitted: 23 Mar 2016
Last updated: 7 Dec 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264.
PubMed 32453377

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
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All Duchenne Muscular Dystrophy trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov ID: NCT02740972
Acronym / sponsor ID: NS-065/NCNP-01-201
Conditions: Duchenne Muscular Dystrophy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: NS Pharma, Inc.Industry (pharma, biotech, devices)
Collaborators: Nippon Shinyaku Co., Ltd., Cooperative International Neuromuscular Research Group, Therapeutic Research in Neuromuscular Disorders Solutions
Study start: Dec 2016 (actual)
Primary completion: Mar 2018 (actual)
Study completion: Apr 2018 (actual)
First posted: 15 Apr 2016 (estimated)
Results: Posted 12 Jul 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Patients completing the 24-week study will be eligible for an open-label extension study.

Safety will be assessed through the collection of adverse events (AEs), blood and urine laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.

Serial blood samples will be taken at four of the study visits to assess the pharmacokinetics of the study drug.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
NS-065/NCNP-01 40mg/kg	Experimental	Drug: NS-065/NCNP-01Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 40mg/kg dose once a week for 24 weeks
NS-065/NCNP-01 80mg/kg	Experimental	Drug: NS-065/NCNP-01Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 80mg/kg once a week for 24 weeks
Placebo	Placebo comparator	Drug: PlaceboTwo patients in each of the dose groups will be administered placebo as an intravenous infusion once a week for 4 weeks followed by 20 weeks of open label treatment

Who may qualify

Ages: 4 to 9 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

Male ≥ 4 years and <10 years of age
Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame;
Able to walk independently without assistive devices;
Ability to complete the time to stand, time to run/walk and time to climb assessments;
Stable dose of glucocorticoid for at least 3 months

Key exclusion criteria (8 listed)

Acute illness within 4 weeks prior to the first dose of study medication;
Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
Severe allergy or hypersensitivity to medications;
Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
NS-065/NCNP-01	Drug	NS-065/NCNP-01 40mg/kg, NS-065/NCNP-01 80mg/kg
Placebo	Drug	Placebo

Primary outcome

Incidence of Adverse Events as Assessed by CTCAE v4.0.
Time frame: 24 weeks of treatment
Treatment emergent adverse events (TEAEs) were summarized for Period 1 by comparing low dose to high dose to placebo and for Period 2 between the low dose cohort and the high dose cohort. TEAEs were summarized both at the patient level for number of TEAEs, highest severity, relationship, action and outcome and at the TEAE level (summarizing events) by organ system and preferred term TEAE as well as severity, relationship, action and outcome. The Medical Dictionary for Regulatory Activities (MedDRA) version 20.1 was used and the Common Terminology Criteria for Adverse Events (CTCAE) grading.
Dystrophin Production by Western Blot
Time frame: Baseline and 24 weeks of treatment
Percentage normal dystrophin production in muscle biopsies from study participants at baseline and after 24 weeks treatment was measured by Western blot. To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. Dystrophin protein levels were assessed using standard curves on each gel (range from 0-25% of normal levels) generated by mixing 5 normal control samples with one DMD sample.

12 secondary outcomes

Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity
Time frame: Baseline and 24 weeks of treatment
Dystrophin Production by Mass Spectrometry
Time frame: Baseline and 24 weeks of treatment
Dystrophin Production by Immunofluorescence
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT).
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB).
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Run/Walk 10 Meters (TTRW).
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity.
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Stand (TTSTAND)
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in Time to Stand (TTSTAND) Velocity
Time frame: Baseline and 24 weeks of treatment
Change From Baseline in North Star Ambulatory Assessment (NSAA) Score.
Time frame: Baseline and 24 weeks of treatment

Results and safety

Results posted to the registry on 12 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo (Period 1)	5	5
NS-065/NCNP-01 40mg/kg (Period 1)	6	6
NS-065/NCNP-01 80mg/kg (Period 1)	5	5
NS-065/NCNP-01 40mg/kg (Period 2)	0	0
NS-065/NCNP-01 80mg/kg (Period 2)	0	0

Primary outcome: Incidence of Adverse Events as Assessed by CTCAE v4.0.

Time frame: 24 weeks of treatment. Count of participants. Unit: Participants

Measure	Placebo (Period 1) n = 5	NS-065/NCNP-01 40mg/kg (Period 1) n = 6	NS-065/NCNP-01 80mg/kg (Period 1) n = 5	NS-065/NCNP-01 40mg/kg (Period 2) n = 8	NS-065/NCNP-01 80mg/kg (Period 2) n = 8	Total n = 16
Participants with TEAE	3	4	4	5	7	15
Participants with drug-related TEAE	0	0	0	0	0	0
Participants with CTCAE ≥ Grade 3	0	0	0	0	0	0
Participants with TEAEs leading to discontinuation	0	0	0	0	0	0
Participants with serious TEAE	0	0	0	0	0	0
Death	0	0	0	0	0	0

Population analysed: Within each category, patients were counted only once if they had \>1 event reported during the treatment period.

Primary outcome: Dystrophin Production by Western Blot

Time frame: Baseline and 24 weeks of treatment. Mean (Standard Deviation). Unit: % of normal control levels

Measure	NS-065/NCNP-01 40mg/kg n = 8	NS-065/NCNP-01 80mg/kg n = 8	Total NS-065/NCNP-01 Group n = 16
Normalized to Myosin	5.7 (2.37)	5.9 (4.50)	5.8 (3.47)
Normalized to Alpha-Actinin	5.4 (2.79)	3.7 (2.37)	4.5 (2.64)

12 secondary outcomes are reported in the full results.

Adverse events

Time frame: 24 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo (Period 1)	0 of 5 (0%)	3 of 5 (60%)	0 of 5
NS-065/NCNP-01 40mg/kg (Period 1)	0 of 6 (0%)	4 of 6 (66.7%)	0 of 6
NS-065/NCNP-01 80mg/kg (Period 1)	0 of 5 (0%)	4 of 5 (80%)	0 of 5
NS-065/NCNP-01 40mg/kg (Period 2)	0 of 8 (0%)	5 of 8 (62.5%)	0 of 8
NS-065/NCNP-01 80mg/kg (Period 2)	0 of 8 (0%)	7 of 8 (87.5%)	0 of 8
Total	0 of 16 (0%)	15 of 16 (93.8%)	0 of 16

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32453377Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Paula R. Clemens, MD study chair

University of Pittsburgh

7 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 7 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Jul 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Adverse Events as Assessed by CTCAE v4.0. (time frame: 24 weeks of treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02740972, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Dec 2021.